A closely watched study using gene therapy to treat sickle cell disease cured one patient, a boy in France, researchers reported on Wednesday, a glimmer of hope for a long-neglected disease but one that comes with several caveats.
Results from the clinical trial, which is sponsored by Cambridge, Mass.-based Bluebird Bio, have been dribbling out at scientific meetings and in company announcements since soon after the boy received the gene therapy, in October 2014, when he was 13. The new paper, in the New England Journal of Medicine, provides a “deeper view” of the patient’s disease and recovery, said Bluebird CEO Nick Leschly. “It’s a bit of a megaphone, allowing us to say that gene therapy might lead to a dramatic outcome.”
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Keep me posted.
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Pls keep me updated
I want to subscribe on behalf of my Daughter, who is sickler